Syndax financials, update, call

 Topline data from AUGMENT-101 KMT2Ar patients expected in the third quarter of 2023 –

–Topline data from the pivotal AGAVE-201 trial on track for mid-2023 –

– Two U.S. registrational filings expected by the end of 2023 –

Financial Update and Guidance

In December 2022, Syndax issued 7,840,909 shares of its common stock at a price to the public of $22.00 per share. This includes the exercise in full by the underwriters of their option to purchase up to 1,022,727 additional shares of common stock. As a result, Syndax received aggregate net proceeds of $162.0 million after deducting underwriting discounts and commissions and estimated offering expenses payable by Syndax.

For the first quarter of 2023, the Company expects research and development expenses to be $30 to $35 million and total operating expenses to be $40 to $45 million. For the full year of 2023, the Company expects research and development expenses to be $160 to $175 million and total operating expenses to be $225 to $240 million.

Conference Call and Webcast

In connection with the earnings release, Syndax's management team will host a conference call and live audio webcast at 4:30 p.m. ET today, Tuesday, February 28, 2023.

The live audio webcast and accompanying slides may be accessed through the Events & Presentations page in the Investors section of the Company's website. Alternatively, the conference call may be accessed through the following:

Conference ID: SNDXQ422 
Domestic Dial-in Number: 800-245-3047 
International Dial-in Number: 203-518-9765 
Live webcast: https://www.veracast.com/webcasts/OpenEx/General/SNDXQ4.cfm

For those unable to participate in the conference call or webcast, a replay will be available on the Investors section of the Company's website at www.syndax.com approximately 24 hours after the conference call and will be available for 90 days following the call.

https://finance.yahoo.com/news/syndax-pharmaceuticals-reports-fourth-quarter-210300827.html

Elon Musk to unveil Tesla's 'Master Plan 3' on Wednesday's investor day — here's what to expect

 'Path to fully sustainable energy future' to be unveiled Wednesday, Musk promises

Tesla Inc. Chief Executive Elon Musk teased a "Master Plan 3" for the electric-vehicle maker on Wednesday, nearly a year after he first tweeted about the blueprint.

Musk said earlier this month that the plan's details will be unveiled at Tesla's (TSLA) investor day Wednesday at the company's Texas headquarters. The livestream starts at 4 p.m. Eastern.

"We suspect his plan will tie together" with Tesla's 2016 Master Plan 2, mentioning robotaxis, shared rides to reduce driving miles, "megapack" batteries, and recycling targets, said Chris McNally, an analyst with Evercore ISI.

Tesla could also give investors hints about a new, cheaper EV platform, said Toni Sacconaghi at Bernstein.

"We believe that the most important issue for Tesla going into its analyst day is the status of its next-gen, lower-cost vehicle platform," he said.

A product announcement would be unlikely, but investors would welcome "incremental detail on pricing, offering and especially timing."

Musk tweeted on March 16, 2022, that he was "working on" the plan:

Musk released the second Master Plan in March 2016, promising a few things that have fallen short of reality.

Tesla still does not have an electric bus, for instance, and a basic trim of its cheapest electric vehicle, the Model 3 sedan, starts at $43,500. Tesla said in January that a "next generation" vehicle platform is in the wings.

Part 2 also included some autonomy and car-sharing goals that haven't come to pass.

The first plan, from 2006, outlined Tesla's strategy of starting off with a pricey and highly desirable sports car as a springboard to future more affordable EVs.

Autopilot and Full Self Driving, Tesla's suite of advanced driver-assistance systems for highway and urban driving, are under probes from several state and federal agencies. Tesla is currently going through a FSD recall.

Tesla "robotaxis," shared driverless cars, are also not yet available.

Consumer Reports recently ranked Autopilot "middle of the pack" in autonomy, bestowing higher marks on Ford Motor Co.'s (F) BlueCruise and on General Motors Co.'s (GM) SuperCruise.

"Tesla hasn't changed Autopilot's basic functionality much since it first came out, instead just adding more features to it," Consumer Reports said.

For all ADAS, Consumer Reports cautioned that driver attention is needed at all times, saying that "cars that can truly and safely drive themselves remain a long way off."

Shares of Tesla have lost 29% in the past 12 months, compared with losses of around 9% for the S&P 500 index.

https://www.morningstar.com/news/marketwatch/20230301251/elon-musk-to-unveil-teslas-master-plan-3-on-wednesdays-investor-day-heres-what-to-expect

Santhera shores up funding ahead of vamorolone verdict

 Swiss biotech Santhera has raised CHF 22 million ($23.5 million) in bridge financing that it says will allow it to fund its operations through to October, when the FDA is due to deliver a decision on its review of Duchenne muscular dystrophy drug vamorolone.

Existing investor Highbridge Capital is providing the new funding, which includes a purchase of 3 million shares in Santhera for CHF 22 million.

Santhera completed its rolling license application for vamorolone in December, which was accepted by the FDA in January, kicking off a review that could lead to the launch of the drug in the US in the fourth quarter.

Vamorolone is also under review in the EU after being filed with the EMA last October, so approval decisions are expected towards the end of the year in both the US and Europe, and the company has said it wants to launch immediately after approval.

Santhera had sought a priority review of vamorolone, but in the end the FDA opted for a standard, 10-month review, although it will not require an advisory committee meeting on the drug.

At the same time, the Swiss biotech has launched a strategic review of its business to gauge the potential of vamorolone in additional indications – such as asthma, inflammatory bowel disease, rheumatoid arthritis, and multiple sclerosis – and possibly licensing out the drug to a commercial partner.

Also potentially up for partnering deals are Santhera’s two other therapies, lonodelestat for cystic fibrosis and Leber’s hereditary optic neuropathy (LHON) therapy Raxone (idebenone).

Dario Eklund, Santhera’s chief executive, said the new funding should provide “liquidity” through to the FDA decision.

“Discussions are ongoing with various parties on potential opportunities to access the resources required to ensure an effective roll-out of vamorolone once approval has been secured,” he added. “We will look at these opportunities with our board of directors in the coming weeks and months.”

Vamorolone was licensed from US biotech ReveraGen BioPharma in 2020, after Santhera abandoned development of idebenone for DMD. If it does get approved, it will mark a turnaround in fortunes for Santhera, which was forced to slash staff after idebenone failed a phase 3 trial in DMD in 2020.

In the phase 2b VISION-DMD study, vamorolone had positive effects on mobility measures like the six-minute walk (6MWT) test and time to stand (TTSTAND) velocity compared to placebo at 24 weeks, with the benefits maintained out to 48 weeks, in DMD patients aged four to seven.

Trials of the drug are ongoing in younger DMD patients aged two to four, as well as the seven to 18 age bracket, and also in patients with Becker muscular dystrophy (BMD), which is less common than DMD and tends to cause milder symptoms.

https://pharmaphorum.com/news/santhera-shores-funding-ahead-vamorolone-verdict

Pfizer’s RSV shot clears FDA adcomm; now it’s GSK’s turn

 An FDA panel of experts has recommended approval of Pfizer’s respiratory syncytial virus (RSV) vaccine in a pair of votes, neither of which was a resounding win.

The Vaccines and Related Biological Products Advisory Committee (VRBPAC) voted by 7 to 4 with one extension that the RSVPreF shot was safe, and by the same margin that it was effective when used to reduce RSV-related lower respiratory infections (RTIs) in people aged 60 years and over.

The dissenting voices raised concerns about a link between the vaccine and Guillain Barré syndrome, a rare disorder in which the immune system attacks nerves that was highlighted in FDA reviewer documents published ahead of the meeting.

It is a rare condition, affecting up to 3 people per 100,000 in the over 60 age bracket, but there were two cases in Pfizer’s ongoing RENOIR study of its vaccine, which enrolled around 37,000 subjects.

Some advisors also said they would like to see the final data from the vaccine first, particularly as the number of RTI cases in RENOIR is fairly low.

There was also debate about the population of patients in the trial, with some panellists arguing that it included too few subjects who were vulnerable to RSV complications, such as those with heart disease or chronic obstructive pulmonary disease (COPD).

Pfizer presented interim results from RENOIR last August, showing 66.7% efficacy in preventing RSV-related RTIs with two or more symptoms, and 85.7% efficacy against three or more symptoms.

The FDA will now have to take its experts’ advice as it works towards completing its review of RSVPreF, which has the proposed trade name Abrysvo. It is expected to make a decision in May.

Currently, there are no approved vaccines for RSV on the market, and Pfizer is in a close race with GSK to bring the first one to market.

GSK’s rival jab now in the spotlight

GSK’s RSVPreF3 jab will come under the scrutiny of the VRBPAC later today, and showed similar, but slightly higher, levels of efficacy in its AReSVi-006 trial reported in October. According to the FDA briefing document, it saw one case of Guillain Barré syndrome thought to be related to vaccination in around 27,000 subjects.

The rivalry between the two pharma companies has also spilled over into the courts. Last June, Pfizer filed a lawsuit seeking to invalidate three patents covering GSK’s vaccine in a UK court.

RSV causes around 177,000 hospitalisations in the over-60s in the US each year and 29,000 deaths, and analysts have predicted that vaccines could generate a market worth several billion dollars a year worldwide.

“In older adults, RSV can result in serious illness, hospitalisation, or even death, so there is a significant need to protect this at-risk population,” said Annaliesa Anderson, chief scientific officer of Pfizer’s vaccine division.

“We are encouraged by the outcome of today’s VRBPAC meeting, as it is a testament to the strength of our science and dedication to bringing this important vaccine candidate to the market,” she added.

https://pharmaphorum.com/news/pfizers-rsv-shot-clears-fda-adcomm-now-its-gsks-turn

Aravive: Orphan status for pancreatic cancer therapy

 Aravive, Inc. (Nasdaq: ARAV, “the Company”), a late clinical-stage oncology company developing targeted therapeutics to treat metastatic disease, today announced that the U.S. Food and Drug Administration (FDA) has granted Orphan Drug Designation (ODD) to batiraxcept for the treatment of pancreatic ductal adenocarcinoma cancer (PDAC).

The FDA’s Office of Orphan Products Development grants ODD status to a drug or biological product to prevent, diagnose or treat a rare disease or condition affecting fewer than 200,000 people in the USA. Companies that are granted ODD are eligible for incentives, including tax credits for qualified clinical trials, exemption from user fees and up to seven years of market exclusivity after approval.

https://finance.yahoo.com/news/aravive-announces-fda-orphan-drug-210500559.html

Train car carrying 30,000 gallons of propane derails in Florida

 A train car carrying roughly 30,000 gallons of propane was among six that derailed in Florida Tuesday in a scene first responders compared to a “minefield.”

The six train cars fell off the tracks as a Seminole Gulf Railway train rode through Sarasota County, News Channel 8 WFLA reported.

The other five cars that tumbled over were carrying sheetrock.

“It looks like a minefield, honestly,” Southern Manatee Fire Rescue Chief Robert Bounds told the local NBC affiliate station. “The cars are on their side. [The train] was in motion when it happened, so it wasn’t just a tip-over, they really dug in and the tracks are pulled up and torn up.”

The propane tanker car managed to stay intact during the partial derailment and didn’t show signs of any immediate leakage, Bounds said.

“As of right now, there is no real emergency. We’re standing by in the event that there becomes one,” he said. “Right now, the tanks are all sealed, they’re not leaking, there’s no evidence of any damage to them that would cause a leak so we’re just monitoring the situation.”

One of the cars was a tanker of propane, while the five others were carrying sheet rock.

Southern Manatee Fire Rescue

Workers will need to siphon the propane out of the tanker in order to move the train car upright and clear the tracks. That process is where things have the potential to go wrong, Bounds said.

“The real potential will be when they come to offload the product,” the fire chief said. “That will be when we really have to monitor the situation very closely to ensure there’s no actual leakage.”

Hazmat teams are monitoring the tanker from above using drones and on the ground with thermal imaging technology.

Bounds said the remediation efforts could take one to three days and surrounding buildings could likely be evacuated while crews work.

“We want to ensure everybody’s safety first and foremost so we will probably evacuate the buildings up to the front on 15th Street just for however long it’s going to take them to offload the product,” he said.

The area near the tracks includes several commercial buildings where several hundred people work.

The propane must be removed in order to turn the train car upright and clear the tracks — which could take multiple days.

WFLA

The section of the tracks where the train cars derailed is also about 700 feet from a homeless encampment. Workers have spoken to the individuals about the situation.

It’s unclear what caused the train cars to flip off the tracks, Bounds said. No one was injured.

The partial derailment comes weeks after a train carrying toxic chemicals derailed in East Palestine, Ohio on Feb. 3 — worrying residents about the potential long-term health effects from the toxins released.

https://nypost.com/2023/02/28/train-car-carrying-30000-gallons-of-propane-derails-in-florida/

China cryptically warns Elon Musk about sharing posts on COVID lab leak theory

 China is warning Twitter CEO Elon Musk against sharing posts that promote the lab leak theory of the coronavirus, suggesting that such commentary could hurt Tesla’s relationship with the company’s second-largest market.

The cryptic warning came on a social media post by the state-run Global Times newspaper.

The writer was reacting to Musk commenting on a tweet that mentioned the Department of Energy’s conclusion that COVID-19 originated at a lab in Wuhan, China. 

The original tweet, from the account “Kanekoa The Great,” questioned whether Dr. Anthony Fauci, former director of the National Institute of Allergy and Infectious Diseases, was involved in the development of COVID-19 because he had funded “gain-of-function research at the Wuhan lab.“

“He did it via a pass-through organization (EcoHealth),” Musk responded, referring to the nonprofit group that was awarded nearly $8 million in federal research grants to study bat coronaviruses in China. 

Elon Musk was warned by China, in the Global Times newspaper, that he could be “breaking the pot of China” regarding his lab leak theory of the coronavirus.

AP

The Global Times warned Musk that he could be “breaking the pot of China.” The saying is similar to the expression “to bite the hand that feeds you,” according to CNBC’s Eunice Yoon, who was the first to report on the warning.

As noted by the outlet, the electric vehicle maker maintains a factory campus in Shanghai – China is the company’s second-largest market.

FOX Business has reached out to Musk and Tesla for comment.

A Department of Energy report concluded that COVID-19 most likely leaked from a lab in Wuhan, China.

REUTERS/Thomas Peter/File Photo
The Department of Energy has recently concluded that COVID-19 most likely came from the Wuhan lab, according to the classified intelligence report cited by the Wall Street Journal.

The so-called lab leak theory was widely dismissed as a conspiracy or “fringe” theory. It was labeled as “misinformation” by Democrats and major news outlets, and social media companies in the early stages of the pandemic suppressed the theory from being circulated.

https://nypost.com/2023/03/01/china-warns-elon-musk-after-covid-lab-leak-comments/